RARE - Earnings call transcript Q1 2024 | Ultragenyx Pharmaceutical Inc.

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Operator

Good afternoon, and welcome to the Ultragenyx First Quarter 2024 Financial Results Conference Call. [Operator Instructions] It is now my pleasure to turn the call to Joshua Higa, Vice President of Investor Relations.

Joshua Higa

Thank you.

We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com.

Joining me on this call are Emil Kakkis, Chief Executive Officer and President, Erik Harris, Chief Commercial Officer; Howard Horn, Chief Financial Officer; and Eric Crombez, Chief Medical Officer. everyone statements. today's remind be we to forward-looking making like that call, during will I'd are to statements results These subject may our uncertainties differ actual and materially. certain risks and latest the discussed risk Please refer in to our SEC factors filings. now to turn the I'll over Emil. call

Emil Kakkis

February, and our we data afternoon, cognition key good is This UXXXX was in WORLDSymposium we've of of long-term gene a treatment this clinical with the The that programs, and we're reduction rapid correlated of the development. positive focused meaningful syndrome. the CSF multiple improved lot presented this the results and biomarker and meeting cognitive data of Josh, years heparin and that year. data sustained execution harvesting therapy from everyone. Thanks, year in exciting showed sulfate across At already resulted Sanfilippo our in long-term shared

as workshop other participated leaders support heparan of by therapies This a the FDA CSF Marks and gene Setrusumab. to Reagan-Udall surrogate Peter sulfate medicines. support working Foundation. these in the companies this also approval biomarker advocates, on on use benefit The a all precision enable working in and biomarker to diseases MPS data sulfate as and really accelerated of in for We together Shifting recognizing endpoint cell biomarker profound approval and companies patient hosted MPS a be overwhelming the neuronopathic a type would to to the communities and of body the discuss representatives, the diseases. scientists brought on workshop supporting heparan a FDA to industry

study that Osteogenesis last that and Phase Phase led data we accelerated GTX-XXX announced in Phase data Imperfecta. announced patients. enrollment X clearly we the we Cosmic interim Angelman the interest to Syndrome. investigators late weeks two completed we've week, data data body Angelman our interim development study of in enrollment our trajectory of was for presented GTX-XXX study X/X strong change in The in The X the compelling study year Orbit of this a in that Phase larger fundamentally the X/X program. positive shared And ago, can from confirmed Just

with magnitude the This no in previously was to we expansion placebo-controlled This syndrome cohorts. And the evidenced the X/X in can history Angelman these or Ongoing dose as greater safety found we similar be domains have successfully long-term all what the managed. cohorts observed most across and than study studies. dose severe cohorts. patients patients, of and be with genetic escalation those resulted demonstrated continuous GTX-XXX profile where been in Phase Bayley deletions both data and there's treatment natural Importantly, valuing improvement sustained effect observed the by the escalation in has the Scale. typically with on is improvement

cohorts and Angelman recent a the early one score, escalation in our What placebo X.X dose after score. almost was a improvement Bayley-X example, clinical study in study we as point year expansion improvement beginning that the year, Bayley-III one XXX in our For their saw the day in only as cohorts. group in at X-point doubled showed cognition dose

domain the what or than of patient day change more sample multiple and all The world improvement or movement hyperactivity and is the Bayley Multi-domain the that changes the of receptor motor we're we for the allowed communication, came domains MDRI more mobility. MDRI, sleep small Angelman study XXX XX around really in individual like at was the We in potentially the and dose in able and of a physicians cohorts. challenging function in across walking better domains. Responder puzzles more population The multiple greater great Further, we in and seeing cohorts the max we videos year The families. least time improvement also day checklist. at brings a Index a of which the and this calmer, in Families were The patients way across of improvements nature look through motor of of also expansion median all transformative behavior resonate achieved at real-world behavior The attentive, improvements therapy. aberrant at path, function also or saw and on improvement majority this four the across early saw and being domains significant solve the in cohorts. provide cognition with the with across and observed cohorts MDRI the This sense XXX. prior statistically point communication the together of up a to to assessments, independence resulted The after four and including of the domain all day in two eating, this across analysis other significant patients of of provide that escalation fleet combination continued two talked the the receptive change assessments group. severity call. charts about more further their development of the across graphs. a behavior XXX a patient of in cognition, facets kids was real supported to sleeping them. showed across improvements heterogeneous navigate aware April cognition,

safety all the of on C multiple classified show resolved and any received the were as in observed the E. this have quickly from events rare, patients Lower that a with Six issue safety Both are extremity Importantly, mild the and no cohort chronic and study in a extended and cohort occurred XX without dosing is weakness loading moderate through the beginning out patients tolerable with the remaining patient all earlier The we has doses A in issues. and study. B in GTX-XXX phase. now events were presented transient profile. that X of well-understood cohorts the completed patients data and

broad over this continue updates safety understanding regulators, from and at only Given manageable, efficacy is with comfortable we issue our current updates. We've with routine efficacy safety We're put the for you typically this candidate product just those this syndrome. route of product all patient the any profile in in treating X meaningful with of for feedback from strong we you strong safety potential Angelman reviewed has comfort be administration weeks, the some these and together, the With see providing call. profile recent heard physicians by Phase a resulted past including expressed on couple and The our where of transform a the treatment development. enthusiasm population patients acceptable and our and don't this might improvements. with are clearly that patients, confident candidate the of analysts, have we'll plan KOLs GTX-XXX treatment to in agreement

on Chief to over an quarter. Officer, our to update that Now I'll led call Harris, turn Erik commercial another successful Commercial the provide our efforts to

Erik Harris

in I everyone since in Thank Kyowa you, to year. last Emil, and been that Kirin good transition for remind Crysvita's responsible driving has afternoon, North April of everyone. America North with in America. revenue I'll the want performance start Crysvita's

Crysvita given new patients the This to in them Crysvita nearly in the penetration encouraging, augment smooth and by transition, generate additional to continued a for room in XXXX. remained field patient help Approximately XXs, to prescribers in U.S. this in implies The ample ensure demand have resulting and from strong maintain additional continuity community adult through the of their start start QX forms. support prescribed has growing. quarter. the is came XX efforts is We XX% adult low forms continue physicians,

process As their seasonality with the is quarter reauthorization year. beginning insurance as some the at providers typical, this worked through of the had patients

are America, demand. where to our the strength we given in Latin full We projections confident revenue lead year commercialization. in the Crysvita of underlying Shifting U.S.

in Our adding patients team quarter XX to XXX therapy Crysvita since approximately on LatAm over delivered new another launch. successful patients reimbursed totaling by

QX than saw more XX% we also revenue Crysvita's and Argentina LatAm drove Brazil of in from uptick significant Mexico. a While XXXX,

X recently the Crysvita adult Mexico's all in We X pediatric versus payer, are the in take excited hurdles. usually value for recognizes to particularly patients pediatric This market about patients. in second they for reimbursement years such the Mexico, which cleared Crysvita approval. LatAm, X and approved for IMSS, pediatric largest about see years largest

now formularies to local expedite is hospital getting reimbursement patients. these on team for Our Crysvita busy

in underlying demand the on calls, As expect earnings mentioned by in we for products. previous driven revenue uneven confident variability our LatAm quarter-to-quarter I growth but remain patterns ordering

new over QX on XXX launch, prescribers Growth pediatric XX reimbursed Dojolvi. to of prescribers remained in adding we of since In forms reimbursed XX% Moving number The start U.S., the to between new strong. patients XX added a approximately continue start XXXX. adult resulting split grow, and therapy, patients. new on in XX% to approximately with patients XX forms and over

approved by been you has Agency. know, European Dojolvi yet not As the Medicines

as named is Approximately an and treated MPS EMEA Dojolvi of increasing from The the So is France, patients XXX demand region, from XX receiving majority across driven request. requests Europe launch region. are within for the by countries XXXX other first the is sales of are important quarter. we MENA Evkeeza. but year under patient countries across

QX treating processes in patients approval. are we through EMEA of XXXX, reimbursement have we As XXX where MPS nearly regular and

launch In after hub last Evkeeza year. Canada's in to started Health Canada, enrolling EMEA countries XXXX. in more our patients We in expect approval we

In private for team Evkeeza. private approval Our is payers authorities Japan XXXX. closely plans. late enrolled January patients approval physician who pricing secure The insurance working XXXX received to about regulatory and in with community the or and XXXX is payer and is we HoFH reimbursement agreements public in patient The reimbursement in these very April. excited secure in through next with early Japan, reimbursement to step also have is underway, the in launch

we receive for the a We a patients in in strong generating robust QX hub, million appropriate of expect in start revenue. the continue XXXX benefits Evkeeza-label launch and we forms was to quarter to have for on XXXX educate $XXX and our patients. as started Overall, Ultragenyx, physicians

our upcoming the results turn that, share underlying for call all strong to in products across delivering for Given With the Evkeeza guidance. revenue our four and XXXX to commercial we the launches, on quarter. confident details our Howard I'll the remain more regions demand financial

Howard Horn

and briefly summarize release afternoon, good our that press in Erik, today. our were earlier I'll financials everyone. Thanks, reported

million Erik XXXX. America, $X and million, including including net year, compared North from As $XXX $XX $XX America. Turkey in of Crysvita to first underlying and meaningfully in noted, America for Europe. Latin sales million from grow continued total contributed patient we and the Crysvita from to quarter the revenue $XX million million reported prior demand North

of in revenue with North tiers royalties reminder, using transition from based royalty. ranging This a going in shifted region and calculated forward, annual is a a revenue XX%. responsibilities the the from to to sales up to KKC the profit net As share American since commercial XXXX on our April mid-XXs

the of year end a range, expect at As low for of royalty quarter first to result, the the revenue started rate to full blended and the our range. be end we the the upper

Crysvita we the $XX With $X revenue royalty growing underlying first meaningfully million. American year. first the the quarter and increasing in throughout demand, expect was rate to increase revenue quarterly Dojolvi was revenue and in the Mepsevii quarter North million

in share. total was Operating $XXX expenses expenses compensation or were the $XXX noncash included $XXX cost stock-based Our $XX net million which $XX SG&A of of and operating expenses first million, In R&D quarter, loss of million. million, of the of first sales per expenses $X.XX million. million included quarter $XX

March cash XX, cash in marketable was operations the used cash, in securities. $XXX and million In million. net first of $XXX quarter, we As had XXXX, equivalents

cash is typically items than it year The larger payment bonuses. a the of of the other annual the first because includes X use of like quarters quarter

also This of balance receivable commercial accounts due timing the our with activity. high ended a receipts from relatively quarter to

what and provided XXXX for from net we be than used operations to million in quarter less is guidance cash the year. our $XXX last remains Importantly, for expected unchanged

from XX% reaffirming also million, revenue between expected from North Specifically, which between and includes million, revenue and and million all of Latin $XXX is cash our XXXX. in regions Turkey product Crysvita expected XX% $XXX Ultragenyx be guidance Crysvita to are ranges. represents America growth revenue million revenue versus $XXX revenue be and to is noncash and We to it Europe. $XXX to and the America Crysvita includes Total royalties all XXXX which forms

between guidance Our and versus versus XX% XX% XX%. to represents XX% which million, revenue range Crysvita be XXXX. to growth million to growth represents expected Dojolvi $XX $XX is X%

the growth countries blend that, respond Dojolvi call in growth countries requests. where Our turn commercialize represent data will Eric an upcoming faster a who and to of our projections patient provide CMO, lower where Crombez, named With readouts. we we update our on to I'll clinical in

Eric Crombez

I data some GTX-XXX other the Thank month, of I already good you, who we will comments shared the everyone. syndrome. Howard, are enthusiasm on patients that so and last treatment to treating exciting transformative reiterate focus most and Emil that went would, my the patient GTX-XXX programs. with our be afternoon, we advocacy potential through organizations for feel the like has a community hearing from to however, that Angelman all of

are We an GTX-XXX the for at important program. inflection point

meaningful clinically is to global and to Phase X The will that clear XXX XXX have be patients. expected and efficacy, Phase are We approximately enroll we study X study randomized placebo-controlled to a startup. transitioning

data including late-stage to this expect We expect pivotal and the endpoints and Disease from will DTXXXX, around Ia. of plan the therapy Shifting therapy with to program, X our X set gene Glycogen initiate We our of Phase meeting an the these Phase Storage finalize investigational has be portfolio. mid-XXXX. Type year. end data the been study gene Phase to the this plans, and that first end from we duration FDA in treatment X large which quarter scheduled the of for

Phase patients patients, treated response demonstrated therapy. randomized, X their year XX expect clinically placebo-controlled patients enrolled see clinically we meaningful All to in of and reductions significant the fifth to in and daily study X/X a that the Phase in follow-up. the with The cornstarch earliest proven meaningful durable statistically of study has DTXXXX

sleep, and for X/X Our cornstarch cornstarch. of helped understand be potential patients missing can to the that during and their in resulting hypoglycemia reducing the great importance dependence Phase have overnight program families on fatal, children. experience which fear GSDIa with And with regarding better especially all the dose a the us

antibody XXX share of expect in our and without enrollment fully treatment the from and treating we during our do of line patient in control Orbit time Phase within data seen X close to Cosmic, results, imperfecta. in patients could XX We completion the have our X patients recently enrolled top improved done also As glucose and next studies. day Phase X/X record not in importantly, overnight to ended monoclonal the with the Phase and We osteogenesis announced over with We of communities. setrusumab, show Orbit been the months. the human Cosmic support efforts up improvement. couple this and I'll strong for plan and in

half We October. of follow-up on expect will I'll of the data closing portion XX additional remarks. of we year. Orbit Phase This data share the from data to this at the X include call now the build to the in last least turn months Emil study second some presented and back to provide

Emil Kakkis

the we GTX-XXX Phase time our for XXXX. of collected X and in will disease the line be in to X Thank our you, this We've plus expected stage data the would that This expect The from gene of year, close Eric. analyzed. made the In share share summarizing And pipeline. we Phase is On updated months quarter, data for the data approximately of expect top of the significant second initiating quickly end around dosed the UXXXX X catalysts we progress after mid-XXXX X data the Later for the long-term six key was Phase rest patient last clinical FDA scheduled advancing the UXXXX, Phase X X meeting Phase half in GSDIa. all some the clinical first second year. half therapy year. year. Wilson the be our of our in with for and to enable to part I'll the by DTXXXX

are the For two first XXXX. or the Orbit by year-end interim the with portion study, planned early of the UXXXX Phase analyses there anticipated X

the If few for by will top or not report equal of the the that final they than analyses would interim a complete visit the months. second Data will patients is a Interim the and to time Committee XX share a at are would analysis p we collect be threshold by followed positive analysis. occur results announced to less a met, later, company not have stringent as The will unless outcome, study Monitoring analyses of prepare XXX. In months not but analyses, a data require immediately positive. the to a formal threshold to first final interim analysis will event and line a

For XXX, near at of filings products those keeping UX the three for or may and syndrome GSDIa year next have with in Imperfecta or we Osteogenesis BLA track, you with readiness Sanfilippo UXXXX over setrusumab with so.

to the base We of growing to transform us your company-defining are With a instructions. at strong and products disease Q&A lives let's our even provide Operator, the builds of on more positions that patients. that, point please rare questions. on move commercial inflection

Operator

Our line Nochomovitz Instructions] [Operator the from Yigal question Citigroup. you. first Thank with comes of

Yigal Nochomovitz

study? one everybody haven't had sort positive up GSDIa much, talked about remind of coming of a a just What how about data. endpoint you pricing the you product? could are the required Could on lines And of but just time reach the that just primary in on the filing of is of you pivotal strategy too for bit prevalence what terms program? thirdly, GSDIa think perspective you're I then and incidence little program this terms you on give it the and this to in

Emil Kakkis

a program, variable GSDIa primary the cornstarch continuous for endpoint utilization. now So is the

so patients and with primary agreed primary That in the be that between treated starch. have utilization control cornstarch comparing for that for placebo the endpoint FDA. of the the And the we'll good also the group. That's reduction decrease glucose requires

go out. have plan transitioning and In with our we terms that manufacturing data discussion the plant, the we lay of to and to are a filing into and strategy, FDA

FDA we'll need are So, the our the have we but path, assuming to it's more transition time when talk of we'll setting it, and filing But it. data detail the to line and positive. on on manufacturing provide the

with commercial an might pricing. the in around X,XXX We the and incident the that there's with patients X,XXX Now prevalence little under territories, in in That's believe U.S. GSDI a be the last estimate. disease. that are

pricing day, And at treadmill haven't a Patients it, a on severe alive. point. we've disease. of study all It are a an is about every We're there this at the desire. thinking disease. We is the from stay set to urgent is trying it that seen but enrollment

impact, pricing of defines degree I So, I date. And what of an think urgent. gene while the have in urgency is GSDIa success prevalence think therapy launches to is

off to, every get about head their want to want I their want They gun I treadmill. glucose. think point day get down. the say, people This at to They treated. the managing put as

about So, there's work we're still more to excited the but obviously potential, do.

Operator

Our next Anupam question with comes from Rama of line the JPMorgan.

Anupam Rama

just about are the with post think GTX-XXX of say, the the feedback divergent feedback Street, KOL month, you your thinking physician Emil, the update or were getting AAN? what get you just last relative misunderstood most or do to, in discussions points you

Emil Kakkis

think Well, of what there's think meaning And Bayley example. that Bayley, I change heard still doesn't KOL the changes difficulty excited about they're are from we that in the meaningful know for feedback they I and the changes. the changes, appreciating of cognition. for some the These

of that's think I So the major ones. one

weakness about extremity resolved the But XX full was of misconception one it's patients was quickly. safety, load. reported. really now moderate I there the that one some through They think lower patient mild. was It out

small relatively thing. a it's So reversible. It's

talked to move And overstating fine, the They if went an change to Phase things. the not it's I of do ahead they X people a and think didn't deal. they ahead. it's say And you regulators ask already. It's schedule all good. said, are going to end still to not KOLs, meaningfulness. anything accepted I think But us

on So a think we're I path.

need people domain say, I normally to magnitude in change seeing and And the exceptional. it aren't speed people disease the we inform change. I people is story. seeing and saw, think what KOLs understand for two these areas see is multiple all. telling something any we're would think might our hope to don't treatment of combination and that that be at the are quite the of the for to keep need treatment more of of that as seeing it we help to I what And And the that one But improvement domains I how data the data what meaningfulness and unusual straight. talk things that this

So we're you for question. the excited And about it. thank

Operator

from Bank comes Securities. Tazeen line Our of of next with Ahmad question the America

Tazeen Ahmad

going timing OI, groups Just or for plan this in the for to going Cosmic scattered? is terms a to that as for single read do around Orbit submit and study you they're age the different application your And time? out part it be of it to of think at is

Emil Kakkis

but probably at And over as fortunately, younger will got historically Well, same the think actually time. respond almost both patients point faster. true, I even to the the same studies enrolled

time. So, full data. would same I actually expectation read be the out our on the think both file for studies And range probably that will based to about age

do that. So we're good actually in shape to

the it's think thing. I right

age submit I think part and not the be one it the would youngest. tough to of range

excited in the plan. and with we're our it the So, young to to parallel in have both play getting filing, enrolled, that's patient it get both them puts studies in

Operator

Our the next Wang question of comes from with Gena line Barclays.

Huidong Wang

is you the would trial assumption And going the And doses now ahead could loading enrollment. analyses the the your three single hear C since Is GTX-XXX or Orbit Phase X interim be cognition? before primary quickly year-end? trial same also, will doses design the correctly Or to initial And that cohort the endpoint? to than timing, for be use of complete expectation? that in domain since with exploring earlier did doses I very still enrollment full-loading on FDA. that three loading E. be you first also the you Bayley-X you're What will finalize Regarding

Emil Kakkis

that happen. So the about. It's on likely the of at It well multiple rapidly. tool. validated endpoint most. It's improving looking X, Phase most They're Bayley the proves we're an we've GTX-XXX that is a primary cognition It's integration score things talked the known.

cohort great will the certainly FDA and secondaries. domains ones of to. we've these other choice be, it. plan. we one aware of other could had and Bayley's on me the we With likely. got We'll features. It's cognition all discussions most the think, that doses are same include The these potentially the think through key clear number our testing XXX. we've think I X I X of standpoint doses, think have or But of let the we the regard data Xth we're getting the That's wanted But other X And separated MDRI And the day the cohorts is a I to in cohorts, within a on and B, have if A and just expectation use all dose and then separation. actually the secondaries. by they're there's month. X-month

So, it's a slight difference between the two.

So it's not really out. how spread X versus they're a doses X-month X. over It's really about X just exactly period,

the make how testing will final feel in of material difference think We with patients would looking would data, We decision be be there see out between a all now, just that difference a or like. I either. doing our right But safety to was if to. on agency but behave will just at them what any there were don't we comfortable

depends timing The done April, the the essentially. interim when the on got it fractures hit think we bit little timing, be in timing. Now here for a we would and UXXXX on

next is expectation end the toward Our year. of in the year, early possibly the coming

that really frame. that want there's around doing a not We It's time in and be the factors come it's lot to same just clear of So, interim. changed.

let's it is so December, December, And say, in also happening January.

that. we're lot not on. a going But really of there's So changing things other

it, then If And prepare DMC then then it they would clean hits data, could one. to positive, is we p-value it in process the next it less for would the get it go will the that end If early takes in January year, it. before be stringent because then early would less time here. of analyze have standard. hear to it, than next lease may mind for think than expectation our through Keep that positive XXX, year. opportunity we'll not But set and it. we sure. to review the I about DMC and meeting it's happen begin on it's We actual

X.XX. is that's that's may to miss you. result, could for So it great quite not mean be the but But think not Hopefully, because much timing. a it clear I

Operator

comes question line Dae of Next Ha the Gon Stifel. with from

Dae Gon Ha

the progress. the straight questions and away. bundle on Congrats two I'll ask just

just press in the obviously or are on So, in up you for on design going some regulatory trial differences XXX Can the like earlier provide what But the terms GSDXa. to since even they other half, had of duration? bit And question, how a talks or endpoints, about divergences color the mean, conversations when that's FDA. about think do such the I little you've thinking night that and GTX-XXX, meetings the aspect for the enrollment second euglycemia important you're of second release more the might KOL? you bring example, throughout with then an presenting

Emil Kakkis

you so discussions, of to The meaning size like pretty other study. will really and and is to The study European extra haven't decision-making. that the XX-week standard. habit FDA expectation the The importance authorities talk it's not We've or of had the day we to preliminary review what products to it's this just any. be The the XXX the drive design dominant likely authorities had the with going normal is Japanese view you're talk about we're doing. it's

choices. where standard think expect don't be actually were customize I a don't there's lot I differences. to I we other be problem pretty differences going some with them. much going of are think have to statistics these design, I of And be think universe. to think programs If don't which trial, to going But piece I plan any necessary. I for in And is each basic done that's XXX on that a So course, there patients. can, randomized there problem. hard-wired region, the endpoints XXX, we think or there's the

anything. what think and We're straightforward, feedback. of if statistics can of to rest don't the it a is I positioning, there we're more and course, So But pretty will that though proposing be be going about is be problem. that comfortable really

Erik, going FDA's CGM sleep with during monitor can night what between said, high, those did presenting you And right? most for at we our I any patients been the impressed and the the and glucose, the see the watch at night GSDIa and stabilize. doing, the in can starch low right? we've looking the where and forward. will we'll program, that of With take choices CGM the longer, the regard the to time That's was monitor, we've position When X/X, I the at turn at Phase look time you As dominate corner likely look and on mostly glucose fraction you were their who their not taking would tracings. been we'll been the data, kids

And releasing so, you knew their livers were glucose. on turning and

pattern night for they look safe. safely them and going their to livers through go that that can are keep that going we're So to the

be at do to I sure looking CGM. we'll So wasn't anything that Eric? think there's else, just you able

Eric Crombez

We setting. challenge the in hospital are controlled fasting maintaining the

will we them monitor overnight a in in, them controlled fast So them setting. and bring

So further of we'll really basis. from large data amount the a a be CGM getting on from support daily that's

Dae Gon Ha

So am XX I an the understanding across the night going you weeks if be every of on untreated and graph, an be basis? to would will, the it across average treated correct? It's versus what high low

Emil Kakkis

doing interval there's glucose their patient, levels. an time where each intensive of monitoring for we're So

ranging We're for the We're doing not We over a how can are between do create an their patient During And target. way had this present patient the you'll in and the control average to we've right, that their then each then chart there's which and that, of graph, but range, that. shows range. the low. time. see those mean. where the more averaging periods, a of talk they're we'll And we'll for take set own time bar about taking high like two over that a each groups, their before where mean they

over tightening of and watching maintaining is and decreasing time, patients. sense we've kind see which hypoglycemia you can no what the of other improving our control, been in up So hyperglycemia

Operator

Kluska comes of Kristen question next with from the Our line Cantor Fitzgerald.

Kristen Kluska

where about for help asked frame kind the get that can please And often help predict readouts We these are Could again, of us And first frame the some the setting for to expectations? that to setrusumab. controllable factors are about. how of advance? then interim less help two in expectations items you we're some sure we what of

Emil Kakkis

we've to expect. I better. I of it be -- what Well, would if reduction to lay our think I very to going would expect similar what Phase be kind X think that seen, down not a we're data

So The a anything, enrolled of only X enrolling we're that. months might think. comfortable are rate, minimum higher we there X of found average I fracture patients XX% reduction. with exposure, a an have patients with If months, very

something it's And would that -- to be you are who consider expect controllable. don't with so fractures, if -- are reduction They see. not enroll patients exactly to have we benefit when threshold to I expect are. that you know we're you'd they get because in very be who eliminating in essentially fracture we patients rates the of wouldn't that But to able would low have a requirement time. and demonstrate necessarily trial, period Those

enrolled, the what type, to think And before. set of you I with don't type uncontrolled which I anything. saw number we replicate of think I Eric, really if I patients the any factors. up ourselves we've have don't see know,

Eric Crombez

patients the with factor we with studies have know fully the there. fractures. mean, are of yes, treatment good would and agree, may you definitely. uncontrollable couple be, I especially in degree I I good fractures really first it X, would arriving say, of But enrolled. setrusumab was and of takes really to before, and Yes, the months, there. in when patients, rate X may is controllable I some there mix the was a X of a effect types enrollment Yes, biggest get initiating you're first unpredictability think the factor

Emil Kakkis

feel and good that more didn't that true it caused active. people that getting probably look the or didn't they felt they but fact fractures, that People also some really like It's worried the -- like was are fracture. are maybe of some kids more

concerned the start they're being are might to fact going and doing actually that active. it's good stronger feel look like doesn't cause It like we about not that are their more his bones and problem great. So a looks

truth So probably the that action actually bone more strain strengthens bone, because the bone that puts the the by are is strengthened actual activity faster the in action. actually

question. you thank for the So

Operator

line of Raycroft with the from comes Maurice Jefferies. question next Our

Maurice Raycroft

ask on I'll Maybe one disease. Wilson's

Just of benefit in frame or what you time benefit? get time insight copper the into assessing for the global when wondering point you're metabolism. the how can drives adequate changes and endpoint And measure

Emil Kakkis

how key with looking and much is urine urine, a copper The out sort of much main is in oozing at the coming the tissues out the sign is Well, is out free letter. two copper a which right? of ways copper coming how of we're

bile, in you detoxifying copper So, have through of out less you if the coming the should get if rid you detox then your urine.

So that's the side the equation. of detox

the On on the [ceruloplasmin other oxidase is side of the copper question, activity]. loading

looking way correctly measuring activity, of which ceruloplasmin on very oxidase ceruloplasmin. be copper loaded at is we'll the So, sensitive a

assays was the sure, sense I [indiscernible]. Eric, add? biomarker two us anything of wasn't a those give will overall else to there So

Eric Crombez

study. Yes. biopsy doing sub We're liver a

This biopsies. for optional, did will in made time opt we've first it do liver is great. really to been but of the that. be the patients majority able So, We it

interesting concentration histopathology So, to be samples. in and those at copper it look will

Maurice Raycroft

just in We X, X of -- think, that Endo, for you're guiding and this the which you've a to and is, longer-term the title I question having quick year. Phase on guiding scheduled got June see to Maybe at half setrusumab. data a you're second

[ENCORE]? presentation Just expect an at wanted should that we expect Or that -- to Endo update? be we clarify, should what in will

Eric Crombez

will be presentation. pure That Yes. ENCORE a

Emil Kakkis

see We'd frankly. to good like news again,

Operator

next from comes of Stanley.' with Morgan Jeff Our question Hung line the

Lee Hung

would take the study Orbit For what interim? [bar be] interim, it stop the what Cosmic confidence if the go that the then was setrusumab early in study, the at the would second for you the point? for early to for you study, study study in have that? And second stopping the scenario, that And stopping you greater for to the with

Emil Kakkis

as interim right would planned now we've at X.XX. The second be

the So that's And final much X.XX. is less tenfold right? stringent, p-value. larger then That's

point, second would in least year is the reason at stringent that that of the have a less everyone think treatment. I So, at

issue less the there. go just months we to the we So, there if of And would have regulatory then sort XX backstop be was X.XX, felt that long.

For randomized it's Cosmic, remember, but open open-label an study, label.

looking And at And the DMC we'll x-rays kids. our of would little be at these the DMC. looking so, are be

stark probably inferior the will an study, will on rates DMC fracture One in is that keep be, treatment. and questions the in study a of the separation is if there not the be obligated kids end to

were criteria separation felt for was groups strong we so at stop. haven't to they [study] be And that hit if during the that on they they a that, looking of will And year. study the the while set could

since they younger finished that of we're ready. be be enrollment, pretty expectation time the study interim other if the doing first -- the should Our is the be by the will the along. we patients one think one think faster one should I respond far hits, and will study, other And the

DMC year the closely And two. be probably and a data it's in a deal lot at remember, the have along a reason that going another there is to you're and study. But action two So pretty So we'll different look old the each with a a will issue, DMC a open is if be take to But to take label, being all fortunately, big bone coordinated have you at the years opportunity determine right? study. chance time that stop look. they has

Operator

from Next Truist. Joon Lee question the comes So with line of

Joon Lee

MBRI would endpoint? it [SEBAR]. wanted strongly if not the would X that be sailed? already neurology department? to For request you an how ship Or are And possible talking [SEBAR], program, be the Phase advocating reviewed And it's has by [SEBAR] approvable the or you to for as GTX Angelman be meeting, to

Emil Kakkis

the Well, we've meetings part doing work We're that And great job. time. group, sciences leader with. Tiffany -- great before and they're in And had under she's we [SEBAR]. a is are good psychiatry the of where actually then actually we've the but to Fortune neurology of that's the group. said whole in we've been

So, I'm and going. in it's we're happy setting with -- very how actually the we're

mantra, and So no at to to -- us this there's fit purpose. not it's that to point. a no [[SEBAR] it's there's really the well-characterized really benefit going department] so really not Sebar or there's neurology It's doing drug, within the

we one. regard With like where is something are that with comfortable it different. very MDRI, MDRI, we we're to but So

used While it the lot is meetings, we [indiscernible] FDA. in was and helped had of conferences. had support lot a a it's I new program it we've methodology for presented And program, of our still and had a

growing I with be think use it as they'll it. we support

MDRI press is single product. to which see way be the prefer. case FDA start a then And key I We same once there, and type of us secondary. and than in randomized in primary in what belief gaining analyzing long particularly it get both. time, at I that And to MDRI understand the put or my want performs may and MDRI FDA put probably validated believe So, our our the as plan better we and it, how a of the support allows manage rather why it's our best to is That endpoint way for we the they'll appreciation run disorder get analysis. way them do a superior in that heterogeneous clinical outcomes. will in fundamentally approach doing new a trial, think sees large more the neurological

Operator

question next of Schwartz Our comes Joseph Leerink from line the Partners. with

Joseph Schwartz

today. quarter. is the Will on Joe on progress for this Congrats This

cornstarch for on one suggests data that the which abstracts, saw us ASGCT the program. benefit last year. shared the We to DTXXXX in the updated So, recently rebounded the data favorably compared

thinking study later may durability this that all, So cornstarch for we expectations, some the you're and the this how how see if pivotal about on provide just your the of change in might if could you wondering comments quarter? treatment production this at

Emil Kakkis

opinion changed much Well, I on very think don't all. at that that's our

dependent the think that does the I way how what is time all therapy the happens actually in therapy the promoter. the They promoter But the so of and doctor the quickly. very down there's secondary we takes reduction expresses noting a the to on little starch stimulating starch the the -- depend in long gene down, down get and gene are [GlucoseXPase] over glucose bit It on get starch. normal run the because to the time the get using is come that manages have factors. we're some to GSDIa expression

are some are. how operates, but we with where there features of interesting So, that we're comfortable

concern time. saw the short expectations we down, XX% We been you But get don't changed period with a doctors a the individual for within yet We But comfortable that we're our haven't think we how We lot something reduction if study. longer on took were it's titrating have good of look, on to what it it seeing, and in like durability. was think over we're dependent more haul. very patients. long highly

Operator

line the TD of Cowen. Werber Yaron from Come with

Yaron Werber

on Yaron. This is for Brendan

data pipeline. of the the the totally of one quick a expect the might I'm somewhat GSDIa me file top that between kind lines But should potentially gene on I this is filings end flux. manufacturing in we transition timing along to in go assume would the might that GSDIa after And line what of year. dropping that similar understand if and you I the I Correct not, more I same therapy Is or both around it the think pricing. for and to fair OTC light early guess, quickly wrong, differences? kind Just us from next similarly? recently be guess this then would year of And manufacturing, you said be for that for on in-house? Or OTC? expect priced you if

Emil Kakkis

exactly predict of timing the BLA, filing. I Well, the to want don't

time with be go filing at, to can during say, runs. submitted filing, those at right, the the We have of have FDA And our they in manufacturing meet for to internally. be transitioned filing. versus running whether let's the to require of example, manufacturing We're sure, we the make

meeting those and filing. [indiscernible] a will the the that timing that need see get of exact impact and And we are factors in straight. to So get data them

out put more we'll a data. little bit So

can will everything have want fast out. to but we bit like so I filings I we're next delay What we at that up OTC it's year going year, that. go. further straight. regard the right end pieces a that's But going some everything being don't in next you to Our I'm look have so, over we we to With will going year. expectation be how it's successful, predict now, be to in is to the to something next a whether it years pieces prediction of don't to if actually more know well, or we front the is all say though late make of that to if likely timing, because not six probably three and us early get have sure -- want get

we severity very pricing, have difference the I'm OTC Phase out. busy I a expensive With regard population OTC RAVICTI, to similar busy, don't So, three happens have how to So, X is including so, in we'll think They're GTX-XXX. be or and be within drug we're to the of and next of there's be sure going patients price right? like once use. sizes a will in very fit lot big which diseases. to a year

cost the get going in justify and any pricing a listening closer haven't could put we now, watching with up the stake assuming to on, right off production, probably and with You drugs. other what's we'll were regard easier patients their ground getting regarding But pricing. we plan We're come in.

Operator

Our question next line comes Sachs. Goldman from the Salveen with Richter of

Salveen Richter

Lydia on Salveen. This for is

you would could the this the what as X just the on FDA meeting and And positive to of plan to to us here? outcome a remind meeting you disclose. another end details plan Street view What Angelman. message post you for with how Phase Just you

Emil Kakkis

approval, outcome as for be taking the So endpoint, that approach positive it the of that statistical ability well the endpoints fillable would as idea. to is forth I we're and get finalized pallet put think essentially what and

most we'll meeting solution, that's to interested and them, very out on that have complete in. With finalizing with So, have and issue and But put a majority endpoints studies, already if dose on agreement regard regimen what to comfort dosing we basic endpoints. notice well clear as messaging, the on the the want while are Street be the a we I set as the the think fact. to duration some will I people

at clear, solve, have there's that would pieces I and finish until of still mean to there agreement be case only we is pretty in we then discussion to announce, time. in we'd that couple which a Sometimes, to announce a but which point final

Operator

Baird. with question the comes next Our of Allen line from Jack

Jack Allen

that rate their the some seasonality the there are you have around to recently as did thinking the annual study? of very for I on as I what KOL rate well. based provide context wanted mentioned to idea, also can Any are they know Orbit? but people a you about patients fracture ask, matching you this placebo in on fracture few And expectations would seasonal it with relates I work helpful. be enrollment affect and the in talked

Emil Kakkis

We any if the did. but don't control season, to way have we

were things affect And So getting into tell that actually one of was fracture Phase the rate. coming X is XX% to trial fracture coming there's process rate trial. a the beginning. in the out because of trial of the of actually in lot screening you And they're a a a between the fractures fracture into of of occur I'll simply thing part the transporting, last year lot It cars. fact, coming increase will study, the the in in like

fracture have to about say fractures. fractures why the you, one year, but think, harder and me, ground right in -- the per is we're you now, threshold are assumed be right that to predict. would we there's is study are The What assuming I So I middle assuming to you expect. of what ask fractures be to people X.XX want you and that? assume Well, don't

safe You always to a assume in the boundary. want boundary, pick you

somewhat potential many Our reduction ample two, XX% than is to could higher, the enrolled? risks higher the traveling? the And study. of they we who's and than happened How will the far be for are expectation they rate dependent more X.XX How fracture much power on be of power those there is higher and synergize it boundary have the type How is and X.XX. we required. are substantially What at have. reduction, But higher the have if you them? OI should

we've place entry a the for criteria one what So, said good in a I X.XX at having with we're least we're in think year. positioning. of But though,

Jack Allen

understand is matched study, the distributed a a component, geographies? global and it's going patient I across I as to be guess just seasonality the

Emil Kakkis

be -- you you small fracture of Probably. the even right? I based remember, it will between randomize that any you blocks, enough always -- actually rate, on to are if they're trial period small out within also should They in time, be enrolled way blocks we'll fracture creating right? the little November in Yes. going right? will with even be low enrolled So stratifying So enrolled, between number are the also the the to point patients March, of majority two be the the patients groups. high fractures

terms a in number window, of I of tight really in all patients. they're say, So enrolled as would seen synchronously is we've pretty

Eric Crombez

help seasonality, randomization. Yes. with are too, And the to randomizing exactly should but to that you keep not one because randomization through balance one, you're trying

could well. help as that So there

Operator

the Next Luca line of Issi question comes RBC with Capital. from

Luca Issi

for that is the actually just on X Phase X X Phase X interim sufficient [Bisphosphonates] in back versus X the age trial Why to you circling should look the question It slightly you're do landscape? OI. placebo? and stat Or difference? And Any placebo Bisphosphonates ask competitive that's Maybe How Amgen's stat to run the latest just to to XX win year-end? on trial data order In question looks them years remind first age. actually Maybe running better versus number in a XX% the in Phase older So you us reduction two, a versus need the one. obviously you versus a year-end? XX where thoughts running in this years FDA reconcile thinking to asking that of we a X. running versus Phase by in Would versus in like a question trial the is there? Cosmic. reticulated by be something see prior fracture the X replicated on hit is differently. scenario the and Can number versus of

Eric Crombez

answer. that I be reconcile better, that is ours would but simple the

a to be running But both confident also I look feel [indiscernible] X guess, trial to story. XX, half in highest X the of study. we Our so able be with was I to will placebo, sides achieved. at what that we're at BMD but X we're you'll position. on that Amgen that X in good their dose months, tested at data was put dosing That their about The they

now, right look will based stronger that. our than theirs just data on So

really is factor to Cosmic, not important regard we're So setrusumab right far, The other factors. real one we're fractures not many is of now, going. are how With the -- the concerned.

fractures might more ability the in to that For example, or the help take would the it's time. then control X.XX X.X greatly hit it less, was it endpoint if group

is it, So X%, you fractures. hit X% enough but have enough need to to

things. and So that's -- synergistic they those other variables how are the two impact

So, we sure. couldn't say for

to the think one I to is [indicate] hit trying whether going first is stringent.

imagine, of reduction they hitting, were open wanted scale you we relatively going to a those case there's it's was and kids think people possibility the on door the off lower high but And high, already fractures children. wouldn't I placebo. and can keep in we were with

already So they're way gone continuing required. you've placebo what's when past --

the right thing think I them, and so do. that's smart, better to for

Operator

showing no questions in back queue. I'm call to would the the now Joshua I closing further over like remarks. for turn to

Joshua Higa

today's This please concludes or you by there are contact questions, Thank ir@ultragenyx.com. call. phone Thank for you. If at additional joining us. us

Operator

concludes Ladies participation. and call. this Thank today's your you gentlemen, conference for

Ultragenyx Pharmaceutical (RARE) 2 May 242024 Q1 Earnings call transcript